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Completed

NCT Number: NCT00457808

Rapamycin Therapy for Patients With Tuberous Sclerosis Complex and Sporadic LAM

The purpose of this study was to determine if rapamycin reduced angiomyolipomata volume in patients with tuberous sclerosis complex or lam.

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Key information

Conditions

Tuberous Sclerosis Congenital Abnormalities Congenital, Hereditary, and Neonatal Diseases and Abnormalities Genetic Diseases, Inborn Hamartoma Hemic and Lymphatic Diseases Heredodegenerative Disorders, Nervous System Immune System Diseases Immunoproliferative Disorders Lymphangioleiomyomatosis Lymphangiomyoma Lymphatic Diseases Lymphoproliferative Disorders Malformations of Cortical Development Malformations of Cortical Development, Group I Neoplasm, Lymphatic Tissue Neoplasms Neoplasms by Histologic Type Neoplasms, Connective and Soft Tissue Neoplasms, Multiple Primary Neoplastic Syndromes, Hereditary Nervous System Diseases Nervous System Malformations Neurocutaneous Syndromes Neurodegenerative Diseases Perivascular Epithelioid Cell Neoplasms

Age range

18 year–65 year

Sex eligibility

All sexes

Study type

Interventional

Phase

Phase 2

Primary location

Cincinnati Childrens Hospital Medical Center

Cincinnati, Ohio, 45229-3039, United States

About this study

The study design was an open label, phase I/II trial of sirolimus for one year followed by one year off therapy.Patients were seen at baseline,at two weeks to four weeks, and at 2,4,6,9,12,18 and 24 months. Angiomyolipomata imaging was performed at all but the two to four week visit. Complete pulmonary function tests and six-minute walk were obtained at baseline,6 or 9 month, 12 and 24 month visits, while simple spirometry only was performed at all other visits.

Who can participate

Healthy volunteers accepted: No

Only the study team can determine whether someone qualifies for participation.

Inclusion criteria

  • Diagnosed with angiomyolipomas and have either Tuberous Sclerosis complex or Lymphangioleiomyomatosis
  • Between the gaes of 18 and 65 years
  • Competency to voluntarily consent
  • Clinically definite diagnosis of tuberous sclerosis or S-LAM
  • Adequate contraception
  • At least one angiomyolipoma of 1 cm or greater in largest diameter

Exclusion criteria

  • Use of continuous supplemental oxygen
  • Concurrent infection
  • Recent surgery
  • Ongoing or planned pregnancy
  • Lactation
  • Use of an investigational drug within the last 30 days of study entrance

Treatment and study plan

Rapamycin, sirolimus

Drug

Primary outcomes

  1. Volume of Angiomyolipomata

Sponsors and collaborators

Lead sponsor

Children's Hospital Medical Center, Cincinnati

Other

Collaborators

  • The LAM Foundation
  • Tuberous Sclerosis Alliance

Registry information

Official study title

Rapamycin Therapy of Angiomyolipomas in Patients With Tuberous Sclerosis Complex and Sporadic Lymphangioleiomyomatosis

Important dates

Study start
2002
Study completion
2006
First posted
Apr 9, 2007
Registry last updated
Apr 9, 2007

OpenTrials presents study information sourced from ClinicalTrials.gov. The official registry record should be consulted for the latest information.

View the official ClinicalTrials.gov record (opens in a new tab)

This listing is for discovery and informational purposes only. It is not medical advice, does not guarantee that a study is recruiting, and does not determine eligibility. Contact the study team and a qualified healthcare professional when considering participation.

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